SCGE

Celebrating Rad Women at the 2025 Rad Awards On July 30, Philadelphia turned out to joyfully celebrate the women who are making our lives much, much better

Congrats to @ahrensnicklas.bsky.social for being named Rad Girl of the Year at the 2025 Rad Awards! Dr. Ahrens-Nicklas was nominated for her groundbreaking work in personalized gene therapy with baby KJ.

Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-awa...

illustration shows poppies that are clear or fuzzy to illustrate preventing versus treating disease or enhancing a trait

Did you know that the illustrations in our resources like CRISPRpedia are free to use for non-commercial, educational purposes? Check out our #SciArt page to see a selection of the amazing work from our designers and artists. Tag us to show us how you're using them! lnkd.in/e_CGXPAz

Gene Editing Technology and Assays SCGE Learn about the technology and assays projects that are part of the Somatic Cell Genome Editing (SCGE) Phase 2 research initiatives.

which will provide essential data on CMC, Critical Quality Attributes, and toxicology of EV based CRISPR gene therapy for IND submission. This project is led by Dr. Mei He.

Learn more at scge.mcw.edu/phase-2-tech... (2/2)

In this project, SCGE researchers aim to optimize and evaluate their recently developed high-throughput extracellular vesicle (EV) loading platform with rigorous quality characterization on EVs and genome editor payload (1/2)

2025 Rad Awards: Dr. Rebecca Ahrens-Nicklas CHOP doctor Rebecca Ahrens-Nicklas is making treatment breakthroughs that are changing lives — and are poised to change the world

​​Congrats to Dr. Ahrens-Nicklas on being nominated for The Philadelphia Citizen’s “Rad Girl of the Year"!

Read more at: thephiladelphiacitizen.org/reebecca-ahr...

A four-state kinetic model captures essential features of Cas12a R-loop dynamics and provides a biophysical framework for understanding Cas12a activity and specificity. (5/5)

The complex multistate landscape of R-loop formation is ortholog-dependent and shaped by target sequence, mismatches, and DNA supercoiling. (4/5)

Here researchers use single-molecule torque spectroscopy to investigate the dynamics and mechanics of R-loop formation of two widely used Cas12a orthologs at base-pair resolution. (3/5)

As an RNA-guided CRISPR endonuclease, Cas12a engages a 20-base pair (bp) DNA segment by forming a three-stranded R-loop structure in which the guide RNA hybridizes to the DNA target. (2/5)

Dynamic basis of supercoiling-dependent DNA interrogation by Cas12a via R-loop intermediates - Nature Communications CRISPR-Cas12a is a programmable endonuclease used for genetic engineering. Here, the authors use real-time single-molecule measurements to show that Cas12a unwinds the target DNA site in dynamic and r...

The sequence specificity and programmability of DNA binding and cleavage have enabled widespread applications of CRISPR-Cas12a in genetic engineering. (1/5)

www.nature.com/articles/s41...

In vivo prime editing rescues alternating hemiplegia of childhood in mice Prime editing and base editing in mouse models and human cells effectively correct mutations in a gene that causes a childhood neurodevelopmental disorder called alternating hemiplegia of childhood. I...

One-shot prime genome editing ("CRISPR 3.0") of the brain in vivo corrected AHC, a rare genetic disorder, in the mouse model. Another big step forward in the genome editing field.
www.cell.com/cell/fulltex...

Future studies in the described human myobundle platform are expected to aid the development of novel Notch-targeted therapies for muscular dystrophies and aging. (4/4)

These studies show that even after a transient, myofiber-specific upregulation of Notch signaling in myobundles, 3D SCs expanded from these tissues can re-form functional "secondary" myobundles containing an amplified SC pool. (3/4)

Here, a 3D tissue-engineered model of human skeletal muscle ("myobundles") is utilized as an in vitro platform for temporal control and studies of Notch signaling. (2/4)

Human Myobundle Platform for Studying the Role of Notch Signaling in Satellite Cell Phenotype and Function Notch signaling is critical for the maintenance and regenerative response of adult muscle stem cells (satellite cells, SCs). Here, engineered human skeletal muscle tissues (“myobundles”) are utilized...

Notch signaling plays a pivotal role in regulating satellite cell (SC) behavior during skeletal muscle development, homeostasis, and repair. The impact of Notch signaling in human muscle tissues remains largely underexplored. 🧪🧬(1/4)

advanced.onlinelibrary.wiley.com/doi/10.1002/...

Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable go.nature.com/4kCD1Y3

graph of progress of various clinical, described in text at the link

Hot off the presses: our reader-friendly 2025 overview of #CRISPR clinical trials! 🧬 Check it out here: ow.ly/mRPl50WneTS

The $20 Million Bet on CRISPR to Cure Rare Childhood Diseases The center will bring custom gene-editing treatments to children.

Gene Therapy News: Plans for the Center for Pediatric CRISPR Cures at the University of California San Francisco were announced today (July 8th). The Center is a collaboration between the Innovative Genomics Institute and the Chan Zuckerberg Initiative.

time.com/7300257/cent...

Our conversation photo and photo of Baby KKJ

A new Pediatric Center for CRISPR Cures builds on the recent saved life of Baby KJ, who expeditiously had bespoke in vivo base genome editing
@jenniferdoudna.bsky.social @doudna-lab.bsky.social
@innovativegenomics.bsky.social
@chanzuckerberg.bsky.social
erictopol.substack.com/p/new-center...

The recent report published by LifeArc and @geneticallianceuk.bsky.social gives several recommendations on how we can #ChangeTheRareFuture and get treatments to people with rare conditions faster.

Read it here: t.ly/Ka7qn

These data reveal a role for untethered RT or the RT domain of PEs in the repair of chromosomal breaks, calling for evaluation of the long-term effect of PEs and retroviral RT in mammalian cells. (3/3)

Here SCGE researchers show that overexpressed RT or PE increases short insertions and diminishes homology-directed repair following Cas9 cleavage at multiple loci in multiple cell lines. (2/3)

The reverse transcriptase domain of prime editors contributes to DNA repair in mammalian cells - Nature Biotechnology DNA repair activity of reverse transcriptase may negatively affect prime editing precision.

Reverse transcriptase (RT) has been shown to play a role in double-strand break repair in bacteria, yet the impact of the RT component of prime editors (PEs) on normal mammalian cellular functions is unclear. (1/3) 🧬🧪

www.nature.com/articles/s41...

Their goal is to guide microscope engineers and biomedical end users toward optimal imaging methods for specific investigational scenarios and to identify use cases in which additional innovations in high-resolution imaging could be helpful.

Imaging 3D cell cultures with optical microscopy - Nature Methods This Review discusses current 2D and 3D microscopy methods for imaging three-dimensional cell cultures and emerging strategies to address key challenges.

3D cell cultures have gained popularity in recent years due to their ability to represent complex tissues or organs. This article reviews the application of 2D and 3D microscopy approaches for monitoring and studying 3D cell cultures.🧬🧪
www.nature.com/articles/s41...

The Ups and Downs of Conducting Groundbreaking Research with Dr. Kiran Musunuru One day after presenting research that led to a first-of-its-kind medical breakthrough, Dr. Musunuru told us that there was so much more to the story.

🎤 OUT NOW: We spoke with Dr. Kiran Musunuru about work he presented at #ASGCT2025 last month. During our first episode of Soundbites of the Annual Meeting, learn about Baby KJ and the behind-the-scenes story of his customized #CRISPR #geneediting therapy. www.asgct.org/publications...

Want to read some interesting gene therapy research? Check out these publications from SCGE consortium research projects. 🧪🧬

Phase 1 Publications: scge.mcw.edu/publications/

Phase 2 Publications: scge.mcw.edu/phase-2-publ...

Characterization of diverse Cas9 orthologs for genome and epigenome editing | PNAS CRISPR-Cas9 systems have revolutionized biotechnology, creating diverse new opportunities for biomedical research and therapeutic genome and epigen...

To expand the CRISPR-Cas toolbox, SCGE researchers characterized a set of type II CRISPR-Cas9 systems from select bacterial genera and species encoding diverse Cas9s, enabling effective genome and epigenome editing for diverse applications.🧬🧪

www.pnas.org/doi/full/10....

YouTube video by U.S. Food and Drug Administration FDA Roundtable on Cell and Gene Therapy

IGI's @urnov.bsky.social today at the #FDA Roundtable on Cell & Gene Therapy. Grateful for an opportunity to share more about using #CRISPR on demand to treat rare diseases!

Watch here: www.youtube.com/watch?v=0qDh...

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CBER Roundtable on Cell and Gene Therapy The Roundtable will provide you with an opportunity to share your thoughts on advancing the field of cell and gene therapy.

🔊 TOMORROW, June 5: Join @fda.gov’s Cell and Gene Therapy Roundtable featuring ASGCT Board Members Terry Flotte, Paula Cannon, and more, on the panel! Watch on YouTube from 9 a.m. to noon (ET).

Do not miss out on this opportunity to hear from top regulatory leaders! www.fda.gov/vaccines-blo...